- The Accelerating Medicines Partnership® in Amyotrophic Lateral
Sclerosis (AMP® ALS) will create the largest open data platform for
ALS research
- Partnership will enable discovery and development of diagnostic
tools, biomarkers, and better treatments
The Foundation for the National Institutes of Health (FNIH)
announces the launch of a public-private partnership to build the
largest data source for amyotrophic lateral sclerosis (ALS) and
expedite the identification of biomarkers and clinical outcome
assessments that would allow earlier diagnosis and accelerated drug
development for people living with the disease. ALS is a
progressive and ultimately fatal neurological disease with few
treatment options and no cure yet.
Managed by the FNIH, the Accelerating Medicines Partnership® in
Amyotrophic Lateral Sclerosis (AMP® ALS) brings together the
resources and expertise of the National Institute of Neurological
Disorders and Stroke (NINDS) at the National Institutes of Health
(NIH), the Food and Drug Administration (FDA), Critical Path
Institute (C-Path), and other stakeholders from academia, life
science companies, foundations, and patient-focused groups to
tackle the tremendous challenges of diagnosing, understanding, and
treating the disease. AMP ALS is supported by a combined commitment
of approximately $60 million.
“ALS gradually robs people of their ability to walk, talk,
eat—and eventually breathe. Patients can’t wait, given the
typical survival time of two to five years after diagnosis,” said
Julie Gerberding, MD, MPH, President and CEO of the FNIH. “AMP ALS
will help define sorely needed diagnostic and treatment targets and
bring hope to patients and their families who are faced with this
challenging disease.” No available treatment halts or reverses the
progression of ALS.
People with lived experience of ALS, including individuals and
families affected by the disease, caregivers, and those at high
genetic risk for developing ALS in the future, are providing their
perspectives throughout this five-year project. Their insights into
the most urgent needs of people with ALS, or at risk for developing
ALS, are helping to shape the project’s research focus accordingly,
and they will continue to be involved in this collaborative
initiative.
“Working collaboratively, through AMP ALS, the partners can
advance efforts to understand what triggers ALS and discover new
targets for effective treatments, as well as identify biomarkers
that can be used to predict whether promising interventions have
the intended effects in people,” said Walter Koroshetz, MD,
Director of NINDS. “Most of all, we are grateful for the
significant contributions of people with lived experience who
provided so much time and effort into the development of this
program, and we greatly appreciate their continued
participation.”
The AMP ALS research team will collect and centralize both
current and future ALS datasets in a cloud-based, openly accessible
knowledge portal. With access to these collective resources and by
capitalizing on existing biosamples as well as clinical data,
including that from digital health technology, researchers will
focus on identifying and validating biomarkers and therapeutic
targets specific to ALS. They will also address the need for new or
modified clinical outcome assessments, including patient-reported
outcomes.
Currently, the average time to diagnosis is 12 months.
Biomarkers are crucial to earlier diagnosis of disease, potentially
before symptoms appear, allowing for possible interventions and
improved quality of life. In addition, validated biomarkers will
help monitor disease progression and patients’ response to
treatment, improve clinical trial design, and potentially lead to
more effective, personalized drug treatments.
ALS, also known as Lou Gehrig’s disease, causes degeneration of
the nerve cells in the brain and spinal cord that control voluntary
muscle movement. As the nerve cells die, the brain can no longer
send signals to muscles, progressively taking away an individual’s
ability to move. Cognitively, most individuals with ALS usually
remain able to reason, remember, and understand their loss of
function, often leading to anxiety, depression, and tremendous
stress on the entire family. It is estimated that ALS affects about
30,000 people in the United States, with 4,000 to 6,000 new cases
diagnosed each year.
“I got involved with AMP ALS as co-chair because I believe in
the importance of data and collaboration. The data set that we are
creating with AMP ALS will be the most comprehensive ever in ALS
research. And the FNIH is bringing industry together with NIH, FDA,
and nonprofit organizations to make it happen,” said Daniel
Doctoroff, who is living with ALS and whose father and uncle passed
away from the disease. “I am optimistic that I will live to see the
discovery of treatments and biomarkers as the result of this major
initiative.” Doctoroff is the founder of the nonprofit research
foundation Target ALS.
AMP ALS is supported by NINDS, FDA, AbbVie, the ALS Association,
ALS Therapy Development Institute, ALS United, Answer ALS, the
Association for Frontotemporal Degeneration, Biogen, Critical Path
Institute, Eli Lilly, EMD Serono, GSK, Mitsubishi Tanabe Pharma
America, QurAlis, and Target ALS. For a current list, visit this
page. Read what the partners are saying here.
The initiative is a component of the public-private partnership
called for in the Accelerating Access to Critical Therapies for ALS
Act, signed into law by President Biden in December 2021. Multiple
clinical sites across the country have already joined the
NIH-funded Access for All in ALS (ALL ALS) consortium, which will
recruit people with ALS and those at risk for the disease to
collect important clinical data and biosamples.
About the Accelerating Medicines Partnership
Launched in 2014 and managed by the FNIH, the Accelerating
Medicines Partnership® (AMP®) program brings together the National
Institutes of Health, U.S. Food and Drug Administration, biomedical
and life science companies, nonprofits, patient-focused groups, and
other organizations to transform the current model for developing
new diagnostics and treatments. Using cutting-edge scientific
approaches and broad sharing of research data, all AMPs seek to
improve understanding of disease pathways, facilitate better
selection of targets for drug development, and streamline processes
for bringing new treatments to patients. AMP ALS is the latest
initiative in a portfolio of AMP projects expediting discovery
around Alzheimer’s disease, Parkinson’s disease, schizophrenia,
common metabolic diseases, heart failure, autoimmune and
immune-mediated diseases, and rare diseases. To learn more about
AMP, visit fnih.org/AMP.
About the Foundation for the National Institutes of
Health
The Foundation for the National Institutes of Health (FNIH)
builds public-private partnerships that connect leading biomedical
scientists at the National Institutes of Health (NIH) with their
counterparts in life sciences companies, academia, patient
organizations, foundations, and regulatory agencies (including the
Food and Drug Administration and European Medicines Agency).
Through team science, the FNIH solves complex health challenges and
accelerates breakthroughs for patients, regardless of who they are
or what health threats they face. The FNIH contributes to the
development of new therapies, diagnostics, and potential cures;
advances global health and equity in care; and celebrates and helps
train the next generations of scientists. Established by Congress
in 1990 to support the mission of the NIH, the FNIH is a
not-for-profit 501(c)(3) charitable organization. For more
information about the FNIH, please visit fnih.org.
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version on businesswire.com: https://www.businesswire.com/news/home/20240520689793/en/
Melanie Doupé Gaiser Ruder Finn, on behalf of the FNIH
212-593-6459 Melanie.Gaiser@RuderFinn.com